an efficacy and safety study of alirocumab in children and adolescents with homozygous familial hypercholesterolemia

Conditions: Hypercholesterolemia

Summary: primary objective: to evaluate the efficacy of alirocumab (75 or 150 milligrams \[mg\] depending on body weight \[bw\]), administered every 2 weeks (q2w), on low-density lipoprotein cholesterol (ldl-c) levels at week 12 of treatment in children and adolescents with homozygous familial hypercholesterolemia (hofh) of 8 to 17 years of age on top of background treatments. secondary objectives: * to evaluate the efficacy of alirocumab after 24 and 48 weeks of treatment on ldl-c levels. * to evaluate the effects of alirocumab on other lipid parameters (eg, apolipoprotein b \[apo b\], non-high density lipoprotein cholesterol \[non-hdl-c\], total cholesterol \[total-c\], high density lipoprotein cholesterol \[hdl-c\], lipoprotein a \[lp (a)\], triglycerides \[tg\], apolipoprotein a-1 \[apo a-1\] levels) after 12, 24, and 48 weeks of treatment. * to evaluate the safety and tolerability of alirocumab up to 48 weeks of treatment.

the study duration was up to 62 weeks, which included (if needed) a run-in period of up to 4 weeks, a screening period of up to 2 weeks, a treatment period of up to 48 weeks, and a follow-up of 8 weeks.

Eligibility criteria:
inclusion criteria : * participants genetically diagnosed with hofh. * participants treated with optimal dose of statin +/- other lipid modifying therapies (lmts), or non-statin lmts if statin-intolerant at stable dose(s) for at least 4 weeks. * a signed informed consent indicating parental permission with or without participants assent. * for participants on apheresis, currently undergoing stable ldl apheresis therapy prior to the screening visit (week -2) and had initiated apheresis treatment for at least 6 months. exclusion criteria: * participants with ldl-c \<130 milligram per deciliter \[mg/dl\] (3.37 millimoles per liter \[mmol/l\]) obtained during the screening period after the participant had been on stable apheresis procedure or lmt (i.e., stable optimal dose of statin ± other stable lmts, or stable non statin lmts in statin-intolerant participants) treatment for at least 4 weeks. * participants with bw \<25 kg. * participants aged 8 to 9 years not at tanner stage 1 and participants aged of 10 to 17 years not at least at tanner stage 2 in their development. * participants with uncontrolled type 1 or 2 diabetes mellitus. * participants with known uncontrolled thyroid disease. * participants with uncontrolled hypertension. * participants who will receive statin de novo during the run-in period. * fasting triglycerides greater than (\>) 350 mg/dl (3.95 mmol/l) at the screening visit. * severe renal impairment (i.e., estimated glomerular filtration rate \<30 milliliter per minute/1.73 meter square) at the screening visit. * alanine aminotransferase or aspartate aminotransferase \>2 \* upper limit of normal (uln) at the screening visit. * creatine phosphokinase \>3 \* uln at the screening visit. the above information was not intended to contain all considerations relevant to a participants potential participation in a clinical trial.

Keywords: pediatrics, homozygous familial hypercholesterolemia, alirocumab, pcsk9 inhibitor, low-density lipoprotein cholesterol (ldl-c)